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ABSTRACT: The newly available iron chelator deferasirox (Exjade, Novartis) is expected to provide better long-term clinical outcomes and improved quality of life for patients with thalassemia than its predecessor, deferoxamine (Desferal, Novartis), because of its oral tablet form.
We used the Markov model to estimate total additional lifetime costs and quality-adjusted life years (QALYs) gained with deferasirox versus deferoxamine in patients with transfusion-dependent thalassemia. Patients were assumed to be 2 years of age at initiation of chelation therapy. Clinical outcomes in terms of morbidity and mortality from associated complications and life expectancy for the study population were estimated using the databases of the Bureau of National Health Insurance and the Health and Vital Statistics of Taiwan. Treatment costs were based on analyses of health insurance claims for patients with transfusion-dependent thalassemia. Utilities in terms of quality of life were also included in the model. The incremental cost-utility ratio of deferasirox versus deferoxamine was defined by the ratio of the difference in expected lifetime costs to the difference in QALYs. One-way sensitivity analyses were performed to examine the robustness of the results to key assumptions.
Patients treated with deferasirox are expected to experience a lower incidence of associated complications and obtain 2.3 QALYs (discounted) at an additional lifetime cost of US$36,291 per patient (US$15,596 per QALY). Sensitivity analyses showed that the unit drug cost of deferasirox had the greatest impact on the incremental cost-utility ratio. In addition, the incremental cost-utility ratio will increase by delaying the starting age (2 years of age in our study) of chelation therapy.
Compared with infusional deferoxamine, oral deferasirox improved clinical outcomes and quality of life in terms of iron chelation in transfusion-dependent patients with thalassemia at a reasonable cost from a healthcare perspective.
Journal of the Formosan Medical Association 04/2013; 112(4):221-9. · 1.13 Impact Factor
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Hsiu-Hao Chang,
Chia-Hua Chen,
Chih-Hsing Chou,
Yung-Feng Liao,
Miao-Juei Huang,
Ya-Hsin Chen,
Wei-Jen Wang,
John Huang,
Ji-Shiang Hung,
Wan-Ling Ho,
Yung-Ming Jeng,
Mei-Ieng Che,
Hsinyu Lee,
Meng-Yao Lu, Yung-Li Yang,
Shiann-Tarng Jou,
Dong-Tsamn Lin,
Kai-Hsin Lin,
Wen-Ming Hsu,
Min-Chuan Huang
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ABSTRACT: PURPOSE: Neuroblastoma (NB) is a neural crest-derived tumor that commonly occurrs in childhood. β-1,4-galactosyltransferase III (B4GALT3) is highly expressed in human fetal brain and is responsible for the generation of poly-N-acetyllactosamine which plays a critical role in tumor progression. We therefore investigated the expression and role of B4GALT3 in NB. EXPERIMENTAL DESIGN: We examined B4GALT3 expression in tumor specimens from 101 NB patients by immunohistochemistry and analyzed the correlation between B4GALT3 expression and clinicopathologic factors or survival. The functional role of B4GALT3 expression was investigated by overexpression or knockdown of B4GALT3 in NB cells for in vitro and in vivo studies. RESULTS: We found that B4GALT3 expression correlated with advanced clinical stages (P = 0.040), unfavorable Shimada histology (P < 0.001), and lower survival rate (P < 0.001). Multivariate analysis showed that B4GALT3 expression is an independent prognostic factor for poor survival of NB patients. B4GALT3 overexpression increased migration, invasion, and tumor growth of NB cells, while B4GALT3 knockdown suppressed the malignant phenotypes of NB cells. Mechanistic investigation showed that B4GALT3-enhanced migration and invasion were significantly suppressed by β1 integrin blocking antibody. Furthermore, B4GALT3 overexpression increased lactosamine glycans on β1 integrin, increased expression of mature β1 integrin via delayed degradation, and enhanced phosphorylation of focal adhesion kinase (FAK). Conversely, these properties were decreased by knockdown of B4GALT3 in NB cells. CONCLUSIONS: Our findings suggest that B4GALT3 predicts an unfavorable prognosis for NB and may regulate invasive phenotypes through modulating glycosylation, degradation, and signaling of β1 integrin in NB cells.
Clinical Cancer Research 02/2013; · 7.74 Impact Factor
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Meng-Yao Lu,
Yen-Lin Liu,
Hsiu-Hao Chang,
Shiann-Tarng Jou, Yung-Li Yang,
Kai-Hsin Lin,
Dong-Tsamn Lin,
Ya-Ling Lee,
Hsinyu Lee,
Pei-Yi Wu,
Tsai-Yueh Luo,
Lie-Hang Shen,
Shiu-Feng Huang,
Yung-Feng Liao,
Wen-Ming Hsu,
Kai-Yuan Tzen
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ABSTRACT: Neuroblastic tumors are childhood neoplasms that possess amino acid decarboxylase (AADC) activity and can theoretically be imaged by (18)F-fluorodihydroxyphenylalanine ((18)F-FDOPA) PET, a new diagnostic tool for neuroendocrine tumors. In this study, we explored the accuracy and clinical role of (18)F-FDOPA PET in neuroblastic tumors. METHODS: From 2008 to 2011, patients with tissue-proven neuroblastic tumors receiving (18)F-FDOPA PET at initial diagnosis or during follow-ups were enrolled. The sensitivity and specificity of (18)F-FDOPA PET were compared with those of (123)I-metaiodobenzylguanidine ((123)I-MIBG) scintigraphy and (18)F-FDG PET, using tumor histology as the standard. The maximum standardized uptake value and tumor-to-liver uptake ratio on (18)F-FDOPA PET were measured and correlated with AADC messenger RNA level in tumor tissue. RESULTS: Fifty tumors from 34 patients, including 42 neuroblastic tumors and 8 lesions without viable tumor cells, were eligible for analysis. (18)F-FDOPA PET successfully detected neuroblastic tumors of different histologic types in various anatomic sites, at a sensitivity of 97.6% (87.4%-99.9%) and a specificity of 87.5% (47.3%-99.7%). In tumors with concomitant studies, (18)F-FDOPA PET demonstrated a higher sensitivity than (123)I-MIBG scintigraphy (n = 18; P = 0.0455) or (18)F-FDG PET (n = 46; P = 0.0455). Among the 18 tumors with concomitant (123)I-MIBG scans, 4 tumors with viable cells were (123)I-MIBG-negative but were successfully detected by (18)F-FDOPA PET. The tumor uptake of (18)F-FDOPA significantly correlated with AADC expression (n = 15 nonhepatic tumors; maximum standardized uptake value, P = 0.0002; tumor-to-liver uptake ratio, P < 0.0001). CONCLUSION: (18)F-FDOPA PET showed high sensitivity and specificity in detecting and tracking neuroblastic tumors in this preliminary study with a small cohort of patients and might be complementary to (123)I-MIBG scintigraphy and (18)F-FDG PET. By correlating with AADC expression, (18)F-FDOPA PET might serve as a useful imaging tool for the functional assessment of neuroblastic tumors.
Journal of Nuclear Medicine 12/2012; · 6.38 Impact Factor
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Journal of Clinical Oncology 06/2012; 30(21):e184-7. · 18.37 Impact Factor
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Pediatric Blood & Cancer 01/2012; 59(1):203-4. · 1.89 Impact Factor
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ABSTRACT: The ataxia telangiectasia mutated (ATM) gene plays a major role in repairing the double-strand breaks and maintaining the genome stability. In this case-control study, associations of seven ATM single-nucleotide polymorphisms (rs600931, rs189037, rs652311, rs624366, rs228589, rs227092 and rs227060) with risks in childhood leukemia in a Taiwanese population were investigated. Two hundred and sixty-six patients with childhood leukemia and 266 age-matched healthy controls recruited were genotyped and analyzed. The P-values of the distributions of the genotypic frequencies in the seven ATM polymorphisms were 0.8925, 0.2835, 0.5772, 0.8731, 0.3641, 0.9181 and 0.5071, respectively. The Pvalues of the distributions of the allelic frequencies in the seven ATM polymorphisms were 0.6158, 0.1179, 0.6971, 0.7944, 0.1887, 0.6605 and 0.2747, respectively. Although the results did not indicate that ATM polymorphism is directly associated with childhood leukemia, the gene-gene and gene-environment interactions of ATM with other factors is worthy of further investigation in the future.
The Chinese journal of physiology 12/2011; 54(6):413-8. · 0.56 Impact Factor
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Yung-Li Yang,
Chih-Cheng Hsiao,
Hsuan-Yu Chen,
Kai-Hsin Lin,
Shiann-Tarng Jou,
Jiann-Shiuh Chen,
Te-Kau Chang,
Jiunn-Ming Sheen,
Sung-Liang Yu,
Meng-Yao Lu,
Chao-Neng Cheng,
Kang-Hsi Wu,
Shih-Chung Wang,
Jiaan-Der Wang,
Hsiu-Hao Chang,
Shu-Rung Lin,
Shu-Wha Lin,
Dong-Tsamn Lin
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ABSTRACT: The absence of biallelic TCRγ deletion (ABD) is a characteristic of early thymocyte precursors before V(D)J recombination. The ABD was reported to predict early treatment failure in T-cell acute lymphoblastic leukemia (ALL). This study aimed to investigate its prognostic value in Taiwanese patients with T-cell ALL.
Forty-five children with T-cell ALL were enrolled from six medical centers in Taiwan. Quantitative DNA polymerase chain reaction (Q-PCR) was performed to check the status of TCRγ deletion. The threshold for homozygous deletions by Q-PCR was defined as a fold-change <0.35.
ABD was found in 20 patients [20:45] who had higher incidences of induction failure than those without ABD (P = 0.03; hazard ratio [HR] = 8.13; 95% confidence interval [95% CI] = 1.23-53.77) after multivariate regression analysis. Patents with ABD also had inferior EFS and OS (P = 0.071 and 0.0196, respectively). Multivariate Cox analysis indicated that the association between ABD and overall survival was independent of age and leukocyte count on presentation (P = 0.036; HR = 4.25; 95% CI = 1.10-16.42).
The absence of TCRγ deletion is a predictor of a poor response to induction chemotherapy for pediatric patients with T-cell ALL in Taiwan. Providing patients with T-cell ALL and ABD with alternative regimens may be worthwhile to test in future clinical trials.
Pediatric Blood & Cancer 12/2011; 58(6):846-51. · 1.89 Impact Factor
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Yu-Yin Shih,
Akira Nakagawara,
Hsinyu Lee,
Hsueh-Fen Juan,
Yung-Ming Jeng,
Dong-Tsamn Lin, Yung-Li Yang,
Yeou-Guang Tsay,
Min-Chuan Huang,
Chien-Yuan Pan,
Wen-Ming Hsu,
Yung-Feng Liao
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ABSTRACT: The nerve growth factor (NGF)/TrkA-signaling is necessary for neural development, and its abnormality has been tightly associated with the tumorigenesis of various cancers originated from the nervous system. The characterization of key molecules involved in the NGF/TrkA-mediated neuronal differentiation could pave the way for the development of novel therapeutic strategies against neural malignancy. We have previously demonstrated that calreticulin (CRT) is a favorable prognostic factor highly expressed in primary neuroblastomas (NBs) with a more differentiated histology. In the present study, we found that the level of CRT was enhanced in NGF-stimulated differentiation of PC-12 cells through the extracellular signal-regulated kinase (ERK)-dependent mitogen-activated protein kinase (MAPK) pathway. A deficiency of CRT significantly decreased NGF-elicited neuronal differentiation. Furthermore, overexpression of CRT enhanced neuronal differentiation via simultaneously activating the ERK-dependent MAPK pathway. The Ca(2+)-regulating capacity of CRT was demonstrated to be indispensable for NGF-elicited neuronal differentiation. Intriguingly, the expression levels of CRT and NGF receptor TrkA were highly correlated in NBs with differentiated histology, and the coexistence of CRT and TrkA in NB tumors synergistically predicted a better 5-year survival rate. Together, our present findings delineate a CRT-dependent regulation of NGF-induced neuronal differentiation.
Journal of Molecular Neuroscience 12/2011; 47(3):571-81. · 2.50 Impact Factor
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Yung-Li Yang,
Chia-Cheng Hung,
Jiann-Shiuh Chen,
Kai-Hsin Lin,
Shiann-Tarng Jou,
Chih-Cheng Hsiao,
Jiunn-Ming Sheen,
Chao-Neng Cheng,
Kang-Hsi Wu,
Shu-Rung Lin,
Sung-Liang Yu,
Hsuan-Yu Chen,
Meng-Yao Lu,
Shih-Chung Wang,
Hsiu-Hao Chang,
Shu-Wha Lin,
Yi-Ning Su,
Dong-Tsamn Lin
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ABSTRACT: Despite current risk-directed therapy, approximately 15-20% of pediatric patients with acute lymphoblastic leukemia (ALL) have relapses. Recent genome-wide analyses have identified that an alteration of IKZF1 is associated with very poor outcomes in B-cell progenitor ALL. In this study, we determined the prognostic significance of IKZF1 deletions in patients with childhood ALL. This study analyzed 242 pediatric B-cell progenitor ALL patients in Taiwan. We developed a simple yet sensitive multiplex quantitative PCR coupled with capillary electrophoresis to accurately determine the allele dose of IKZF1, and high resolution melting was used for mutation screening for all coding exons of IKZF1. Twenty-six (10.7%) pediatric B-cell progenitor ALL patients were found to harbor these deletions. Most of the deletions were broader deletions that encompassed exon 3 to exon 6, consistent with previous reports. Genomic sequencing of IKZF1 was carried out in all cases and no point mutations were identified. Patients with IKZF1 deletions had inferior event-free survival (P < 0.001), and overall survival (P = 0.0016). The association between IKZF1 deletions and event-free survival was independent of age, leukocyte count at presentation, and cytogenetic subtype by multivariate Cox analysis (P = 0.003, hazard ratio = 2.45). This study indicates that detection of IKZF1 deletions upon diagnosis of B-cell progenitor ALL may help to identify patients at risk of treatment failure. IKZF1 deletions could be incorporated as a new high-risk prognostic factor in future treatment protocols. To the best of our knowledge, this is the first study to examine the poor prognosis of IKZF1 deletions in an Asian population.
Cancer Science 07/2011; 102(10):1874-81. · 3.33 Impact Factor
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Pediatric Blood & Cancer 06/2011; 57(4):697. · 1.89 Impact Factor
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Pediatric Blood & Cancer 06/2011; 57(4):698-9. · 1.89 Impact Factor
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ABSTRACT: This study investigated the immune response to 2009 pandemic H1N1 influenza monovalent vaccine in children with cancer receiving chemotherapy.
We enrolled 25 pediatric patients. Ten patients younger than 10 years old received two vaccinations and the remaining 15 patients older than 10 years old received one. We checked hemagglutination-inhibition (HAI) antibody titers in sera of patients before and 3-4 weeks after vaccination. Seroprotective titer was defined as HAI antibody titer ≥ 40 and seroresponse as ≥ 4-fold increase in HAI antibody titers after vaccination.
The pre- and post-vaccination seroprotective rates were 52% and 72% (P = 0.24). Sixteen (64%) patients were possibly exposed to 2009 pandemic H1N1 influenza previously, and there was significant association between possible exposure and pre-vaccination seroprotective rate (P = 0.03). Post-vaccination seroresponse rate was 32%, and seroresponse was greater in patients without pre-vaccination seroprotective titer than those with pre-vaccination seroprotective titer (50% vs. 15%, P = 0.07). Children with lymphocyte counts above 1,500/µl during vaccination period had better seroresponse than those with lymphocyte counts below 1,500/µl (P = 0.008). Post-vaccination geometric mean titer (GMT) significantly increased in patients younger than 10 years receiving two vaccinations (pre- and post-vaccination GMT were 21.4 and 60.6, respectively; P = 0.025).
Monovalent vaccine for the 2009 pandemic H1N1 influenza A was found to be partially immunogenic in children with cancer, as evidenced by 32% of seroresponse rate. Immune response can be improved with vaccinations administered to patients whose absolute lymphocyte counts returned to a level of 1,500/µl or higher.
Pediatric Blood & Cancer 03/2011; 57(7):1154-8. · 1.89 Impact Factor
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ABSTRACT: A patient with severe haemophilia B with a glycine-to-valine missense mutation at residue 190 (c25, chymotrypsin numbering) in factor IX (FIX; FIX-G190V or FIX-FuChou) had <1% of normal FIX clotting activity and 36% of normal FIX antigen levels (cross-reacting material- reduced, CRMr). Residue 190 in the C-terminal protease domain of human FIX is highly conserved in mammalian species and the serine protease family, suggesting that it has an indispensable role in protein function. To explore the pathological mechanism by which this mutation contributes to dysfunction of the FIX molecule, we functionally characterised FIX-G190V in vitro and in vivo. Liver-specific FIX-G190V gene expression following hydrodynamic plasmid delivery into haemophilia B mice revealed a 5.7-fold reduction in specific clotting activity compared with FIX-WT (wild type) and a two-fold decrease in plasma FIX-G190V concentration. Pulse-chase analysis demonstrated that FIX-G190V was secreted at a significantly slower rate than was FIX-WT. Purified FIX-G190V and FIX-WT displayed normal calcium-dependent conformational changes as shown by intrinsic fluorescence quenching. The in vivo half-lives of FIX-G190V and FIX-WT were indistinguishable. FIX-G190V was, however, more readily degraded than FIX-WT, especially after being activated by the active form of FXI. The vulnerable sites were mapped to the peptide bonds at Arg¹¹⁶-Leu¹¹⁷, Lys²⁶⁵-Tyr²⁶⁶, Arg³²⁷-Val³²⁸, and Arg³³⁸-Ser³³⁹, which are in the exposed loops of the FIX molecule. Also, failure of FXIa-activated FIX-G190V to bind p-aminobenzamidine indicated an abnormal conformation of the active-site pocket. Thus, the mutation at residue 190 of FIX may result in protein misfolding that affects secretion, clotting function, and hydrolysis.
Thrombosis and Haemostasis 02/2011; 105(4):616-26. · 5.04 Impact Factor
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ABSTRACT: Deferasirox is an oral iron-chelating agent taken once-daily by patients with transfusion-dependent iron overload. However, some patients are unresponsive or unable to tolerate once-daily deferasirox. The current study evaluated whether twice-daily deferasirox treatment showed increased efficacy or tolerability in unresponsive or intolerant patients.
Patients from two Taiwanese hospitals with transfusion-dependent β-thalassemia, including those who showed increasing serum ferritin levels for six consecutive months, with at least one level >2,500 ng/dl, while treated with >30 mg/kg/day of once-daily deferasirox (unresponsive) or developed deferasirox-related adverse events (AEs) at the dosage required to maintain the iron burden balance (intolerant) and were treated twice-daily with the same total daily dose of deferasirox since 2008, were enrolled in the study and evaluated retrospectively by medical record review.
Eighteen patients were included for analysis. A statistically significant median decrease in serum ferritin levels was detected in the 11 unresponsive patients after 6 months of continuous twice-daily deferasirox treatment. Five out of the seven intolerant patients experienced either no deferasirox-related AEs or less severe AEs. The 12 patients from both groups (11 unresponsive, 1 intolerant) who received continuous twice-daily deferasirox for 6 months showed a mild but significant median increase in serum creatinine levels.
Twice-daily deferasirox dosing is effective in iron chelation and improves tolerability in transfusion-dependent β-thalassemia patients who are unresponsive to or intolerant of once-daily deferasirox. Future studies with greater patient numbers will be required to confirm the results reported herein.
Pediatric Blood & Cancer 11/2010; 56(3):420-4. · 1.89 Impact Factor
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Yung-Li Yang,
Shu-Rung Lin,
Jiann-Shiuh Chen,
Chih-Cheng Hsiao,
Kai-Hsin Lin,
Jiunn-Ming Sheen,
Chao-Neng Cheng,
Kang-Hsi Wu,
Shu-Wha Lin,
Sung-Liang Yu,
Hsuan-Yu Chen,
Meng-Yao Lu,
Hsiu-Hao Chang,
Ching-Tzu Yen,
Jing-Fang Lin,
Ying-Hui Su,
Ya-Ping Li,
Chien-Yu Lin,
Shiann-Tarng Jou,
Dong-Tsamn Lin
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ABSTRACT: The classification of B-lineage acute lymphoblastic leukemia (ALL) by specific chromosomal translocations has prognostic implications for risk-directed therapy. Reverse transcription-polymerase chain reaction (RT-PCR) assay is a useful tool for detecting fusion transcripts from common chromosomal translocations of ALL cells.
Multiplex RT-PCR and nested-PCR assays were used to detect ALL-type BCR-ABL1 transcripts of the t(9;22), TCF-PBX1 transcripts of t(1;19), the MLL-AF4 transcripts of t(4;11), and 2 variants of ETV6-RUNX1 of the cryptic t(12;21) in 148 leukemic samples upon diagnosis. The patients received risk-directed protocols of the Taiwan Pediatric Oncology Group-ALL-2002 that consisted of multiple chemotherapeutic agents of different intensities. Event-free survival (EFS) and overall survival (OS) rates were analyzed for genetic abnormalities detected by multiplex PCR and conventional cytogenetic analysis by the Kaplan-Meier method, and compared with the Mantel-Haenszel test. The Cox proportional hazards model was implemented to identify independent prognostic factors for EFS and OS.
In this cohort of Taiwanese children, the relative frequencies of the 4 translocations of B-lineage ALL were 8% with ALL-type t(9;22)/BCR-ABL1, 4% with (1;19)/TCF-PBX1, 2% with t(4;11)/MLL-AF4, and 17.6% with t(12;21)/ETV6-RUNX1. Patients with t(12;21)/ETV6-RUNX1 fusion, hyperdiploidy, and t(1;19)/TCF-PBX1 fusion had the most favorable outcomes, whereas those with the t(9;22)/BCR-ABL1 fusion or t(4;11) and other MLL gene rearrangement had poor prognosis (P<0.001 for EFS and OS). BCR-ABL1, MLL gene rearrangement, and very high-risk group were independent prognostic factors after Cox regression analysis.
The biological factors of leukemia cells are associated with treatment outcomes in childhood ALL. Multiplex RT-PCR assay is an efficient and sensitive diagnostic tool that may improve the ability to accurately and rapidly risk-stratify children with ALL.
Journal of Pediatric Hematology/Oncology 10/2010; 32(8):e323-30. · 1.16 Impact Factor
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ABSTRACT: The relationship between COX-2 gene and childhood leukemia risk is ambiguous. In this study, the association of genotypic polymorphisms in cyclooxygenase 2 (Cox-2) with childhood leukemia were investigated.
A total of 266 patients with childhood leukemia and 266 healthy controls recruited from the China Medical Hospital in central Taiwan were genotyped by PCR-RFLP method. Six polymorphic variants of Cox-2 were investigated, including G-1195A, G-765C, T+8473C, intron 1, intron 5, and intron 6, and the associations of specific genotypes with susceptibility to childhood leukemia were analysed.
The data showed that although there was no difference in the distribution for each genotype of Cox-2 G-1195A, G-765C, T+8473C, intron 1, intron 5, and intron 6, between the childhood leukemia and control groups (p>0.05), the analysis of combined effect for COX-2 G-765C and intron 6 showed that individuals with GC at G-765C and GG or AG+AA at intron 6 present a slightly higher potential for developing childhood leukemia than other groups.
These findings suggest that the C allele of COX-2 G-765C may be responsible for childhood leukemia and may be useful in early detection of child leukemia.
Anticancer research 09/2010; 30(9):3649-53. · 1.73 Impact Factor
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ABSTRACT: Engineered recombinant factor IX (FIX) with augmented clotting activity may prove useful for replacement therapy, but it has not been studied for risk of thrombosis. We used three mouse models to evaluate thrombosis risk associated with the FIX variant FIX-Triple, which has a 13-fold higher specific activity than wild-type FIX (FIX-WT). Protein infusion of FIX-Triple into haemophilia B mice was not thrombogenic, even at a dose of 13-fold higher than FIX-WT. Gene knock-in to generate mice that constitutively produce FIX-WT or FIX-Triple protein revealed that all mice expressed equal antigen levels. FIX-Triple knock-in mice that exhibited 10-fold higher FIX clotting activity did not show hypercoagulation. Adeno-associated viral (AAV) delivery of the FIX gene into mice was used to mimic gene therapy. Haemophilia B and inbred C57Bl/6 mice injected with different doses of virus particles carrying FIX-WT or FIX-Triple and expressing up to a nearly 13-fold excess (1289% of normal) of FIX clotting activity did not show increased risk of thrombosis compared with untreated wild-type mice in a normal haemostatic state. When challenged with ferric chloride (FeCl3), the mesenteric venules of AAV-treated C57Bl/6 mice that gave a nearly five-fold excess (474%) of FIX clotting activity were not thrombotic; however, thrombosis became obvious in FeCl3-challenged mice expressing extremely high FIX clotting activities (976-1289%) achieved by AAV delivery of FIX-Triple. These studies suggest that FIX-Triple is not thrombogenic at therapeutic levels and is a potential therapeutic substitute for FIX-WT.
Thrombosis and Haemostasis 08/2010; 104(2):355-65. · 5.04 Impact Factor
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ABSTRACT: Although pancreatoblastoma (PB) is an extremely rare tumor, it is the most common pancreatic tumor in children. We reported 2 cases of PB treated at a medical center in Taiwan. One was a 3.5-year-old boy who presented with abdominal pain. Physical examination demonstrated abdominal masses. Multiple pancreatic and hepatic masses were noted by magnetic resonance imaging. He underwent surgical resection but tumor could not be removed completely, which was confirmed by the pathology. He received chemotherapy consisting of cisplatin and doxorubicin after the surgery but the tumor progressed rapidly. He died of progressive disease and sepsis. The other case was a 4-year-old boy presented with abdominal pain. Computed tomography showed pancreatic tumor. He underwent surgical resection and pathology showed PB. He received chemotherapy after complete tumor resection. He is disease free till now. Complete tumor resection is the major difference of these 2 patients and is the most important factor affecting the outcome.
Journal of Pediatric Hematology/Oncology 04/2010; 32(3):243-5. · 1.16 Impact Factor
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ABSTRACT: Deciduoid mesothelioma is an extremely rare variant of malignant epithelioid mesothelioma. This report presents the case of a 13-year-old boy with this type of tumor in his pleura, whose initial main symptoms were chest pains and progressive scoliosis. Ensuing chemotherapy, which comprised pemetrexed and cisplatin, yielded good response after 5 cycles. Subsequent radical surgery was carried out and another 3 cycles of chemotherapy were given. The patient has been doing well 2 years after completion of this regime of treatment.
Pediatric Hematology and Oncology 03/2010; 27(2):132-7. · 0.89 Impact Factor
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ABSTRACT: We describe a case of catheter-related bacteremia due to Staphylococcus pseudintermedius in a child with dog exposure. The organism was confirmed as S. pseudintermedius based on 16S rRNA gene sequence analysis and positive PCR-restriction fragment length polymorphism of the pta gene.
Journal of clinical microbiology 02/2010; 48(4):1497-8. · 4.16 Impact Factor
